Hopefully a new treatment for spasticity and pain in MS

"Fast Forward, LLC, the National MS Society's subsidiary devoted to bridging the gap between research and drug development, and Concert Pharmaceuticals, Inc. (Lexington, MA) today announced a new collaboration to fund the preclinical advancement of C-21191, a substance that has been modulated to help improve an experimental therapy with the potential for treating spasticity and pain in MS. These are two common and often debilitating symptoms experienced by people with MS. Fast Forward is committing up to $750,000 to help advance development of C-21191 toward clinical trials in people with MS. ....."

"This is brilliant news. I have been lobbying Pharma for years to take MS-spasticty seriously. I must spend a good 10-15% of my clinical activities managing spasticity and dealing with the side effects of the current agents that are appalling. I am sure that any new anti-spastic drug with a better side effect profile and similar efficacy to current runners will be a block-buster drug. If you have advanced MS and suffer from spasticity you should be cheering and celebrating this news."

I'd keep the term brilliant until a cure is found. As poster above says - this will be years away - I'd guess 10 years before available from Boots. Why should we be thrilled about something is a decade away!

If you had advanced MS Prof G, would you really be that thrilled? Tell us news that will benefit us soon e.g. next year. Surely there must be something to thrill us n the short term!

I assume you are not a baclofen "zombie". There are thousands, no hundreds of thousands, of MS'ers who are on baclofen and are paying the price of cognitive slowing, lack of concentration, falling asleep in the day, etc. because of the side effects that come with the drug. Any promise of a new drug with a potentially better side effect is good news. The unmet needs in MS is massive; developing a new symptomatic treatment doesn't mean that work on disease-modifying therapies is compromised. The unmet need in MS therapies is massive; they all needs addressing. So we should celebrate the news that finally industry is looking at improving MS symptomatic treatment. You may feel differently when you need an anti-spastic treatment to control your spasms, to relieve your pain, to allow you to get a good nights sleep and to do this and allow you to read this blog or read your favourite newspaper column. Quality of life is a complex mix; we need a holistic approach to treating MS and its consequences.

Re: "Sometimes I think MS clinicians don't live in the real world. No wonder MS'ers feel so frustrated."

Are you on baclofen? I suspect not; you would feel differently if you were.

Of course neurologists live in the real world; I prescribe the drug almost every day of my working life. Although its partially effective its side effect profile is awful. Tzanidine, clonazepam, diazepam, dantrolene are not much better. What you forget, or choose to ignore, is that every drug begins its life in the pre-clinical phase. We do not peddle miracles, it takes hard work, and a bit of luck, to get a drug to market. The more drugs we have in preclinical development the better; it is a numbers game the more drugs we test the more likely we are to get a new drug for spasticity. I believe in recognising and celebrating small steps! Rome was not built in a day!

Lisinopril is a drug that is licensed for hypertension and it has an impact on inflammation. The strategy was developed by Larry Steinman's group in Stanford. It may augment other drugs and it has a good safety record. It therefore needs to be tested in clinical trials. The problem will be how to design the trials to test the drug.

I note that an Prof G considered advanced MS to present as EDSS >3.5. This seems to be a purely physical measure? What standard of physicality would you expect at the 'advanced level'? Would a patient be able to complete the 6 minute timed walk test unaided? Is it possible, in your opinion for a patient to drop two EDSS points - assuming a start point of 5.0?

Prof G, please don’t take this personally but I sense you are not seeing the whole picture.

Nobody will dispute your commitment and hard work for the MS community. However, your desire to flag baclofen as a reason for why we should be grateful that a new anti-spasticity treatment is in pre-clinical development is rather feeble.

Sure, industry is exploring new avenues like never before, but this is the same industry that has introduced a range of disputably effective MS treatments that are both highly expensive and dangerous to patients.

I am not on baclofen, but then again, I’m not on any drug for my MS. The MS research field has so far failed me as it has countless other MSers’. I’m happy that more MS drugs are in pre-clinical designing but my reality is that these treatments cannot benefit me presently, yet I continue to deteriorate. I’m sorry but I can’t be as enthused as you are about a treatment that is way off, as time is not on my side. I don’t have that luxury. In fact, I find it depressing that the best thing that may be available in 10 years time is an anti-spastic agent as opposed to a remyelination medicine. It’s a reminder of just how much ground has to be made before anyone currently living with MS is significantly improved. We’re still very much at base camp and the mountain is way too high to surmount. I guess that is the reality.

I agree, Rome was not built in a day, but the progress in MS medical development has been painfully slow considering the achievements in say other areas like cancer, AIDS and various diseases. You hear about people being cured of cancer but never have I heard the same about MS.

I guess that we who live with the disease need to get real. Our MS is here to stay. All the rubbish in the press and charity websites about breakthroughs is misleading considering the time lengths involved in drugs coming to market.

Only this morning BBC 1 showed a programme called “Postcode Lottery” in which they ran a feature on MS and how faceless PCTs are neglecting the needs of PwMS by depriving them of Sativex. I suspect similar news stories will come to surface when this new anti-spasticity drug in pre-clinical testing you’re so excited about comes to NICE’s approval.

Re: "I note that an Prof G considered advanced MS to present as EDSS >3.5. This seems to be a purely physical measure?"

Yes, this is based on the EDSS. The reason for this is that all our natural history of the disease has been collected with this outcome. EDSS 4.0 and above is where the scale becomes a disability scale (affects MS'ers functioning), rather than impairment scale (abnormal neurological examination). In addition, the progress from 4.0 to 6.0 (walking-stick) tends to to be particularly rapid and presages the development of secondary progressive MS.

Re: "What standard of physicality would you expect at the 'advanced level'? Would a patient be able to complete the 6 minute timed walk test unaided?"

Yes, all the that the 6MTW requires is for you to be able to walk for 6-minutes. The outcome or metric is how far you can walk in this period of time. A lot MS'ers using a crutch or crutches can walk for 6 minutes. You need to know that this outcome measure was initially developed for people with lung disease. We don't have much experience with it in MS yet. But this will change; there seems to be a momentum behind the outcome measure.

Re: "Is it possible, in your opinion for a patient to drop two EDSS points - assuming a start point of 5.0?"

It depends on how you got to 5.0. If you got there gradually, i.e. from non-relapsing progressive MS, then going backwards on the scale by 2 points is unlikely. On the other hand if you got there from a relapse or acute deterioration from an infection then moving back 2 points is not an unreasonable expectation.

I am a baclofen"zombie" but I need it to reduce spasticity in my legs and hand in order to hold a pen type or dress myself. Without it simple tasks are very difficult due to fingers curling up and terrible pain. It leaves my cognitive function impaired and sluggish so for me this is good news.

I agree with the statement that MS neurologists aren't living in the real world. You're all doing cartwheels for a drug that won't fix your MS and is more than a decade away from getting to us. That's pathetic.

If disagreeing with those who run this blog is considered as 'trolling' then consider me a troll, but I don't agree with that label. All I'm saying is that it's wrong of us to be excited about this news when all it will do is paper over cracks, and even then it's 10 to 15 years away from completion. I mean, seriously, is that the best MS research can offer?

Look Prof G, I realise you're trying your darndest. I also realise you are merely one of thousands of scientists working hard to figure out the exact nature of MS. Your whole mantra is to manage expectations but it almost feels like we're best off expecting not much at all other than minor treatments that will offer slight benefits. What's more, these drugs are a long time coming.

I wish you'd say in posts of this nature that although pharmas are doing these things, the shame is it's a number of years away from getting to us, but there are other things that will fill in the gap in the meantime. At least that's more optimistic.

All I am saying is that the choice of words used in this post are wrong. The notion that MSers with advanced MS and spasticity should be "cheering and celebrating" for a treatment years away from completion is, frankly, insulting.

It is equivalent of telling somebody you’re thirsty and them giving you a sandwich. It’s a really disproportionate statement and Prof G ought to realise why I have reacted badly to it.

Look, nobody likes bad news. Having to wait for symptomatic treatments that are a long way away is not much reason for either cheering or celebrating. If neurologists feel that we MSers should manage our expectations, then I say that they should learn to manage our praise for some of the things they’re working on. I refuse to do backflips for a drug that, by the time it gets a licence, will be of greater benefit to the next generation of MSers than it may be for either you or I.

All I am saying is that the choice of words used in this post are wrong. The notion that MSers with advanced MS and spasticity should be "cheering and celebrating" for a treatment years away from completion is, frankly, insulting.

It is equivalent of telling somebody you’re thirsty and them giving you a sandwich. It’s a really disproportionate statement and Prof G ought to realise why I have reacted badly to it.

Look, nobody likes bad news. Having to wait for symptomatic treatments that are a long way away is not much reason for either cheering or celebrating. If neurologists feel that we MSers should manage our expectations, then I say that they should learn to manage our praise for some of the things they’re working on. I refuse to do backflips for a drug that, by the time it gets a licence, will be of greater benefit to the next generation of MSers than it may be for either you or I.

Dear "insulted" anonymous. If we were talking 10-15 years ago the situation was nowhere near as good as it is now, with many more treatments available and more in the pipeline. If you knew how difficult it was to get pharma to get interested in treatments for MS and that they now are, is largely due to the intense lobbying by the likes of Prof G you might moderate your views.As it is we all would like there to be a cure now, but the real world doesn't work that way but many of us are doing our damnedest to speed things up.We know it isn't fast enough but we really are trying.

Thanks MouseDoctor2 for all your hard work but sympathy also to Anonymous. It's really hard having SPMS and being expected to behave gratefully and politely all the time. Don't forget a career in MS is easier than having it so give him some slack even if he is grumpy

i was the first guinea pig in the world that reacted to baclofen that was fed to my spinal cord from a medtronics pump. i had amnesia delirium for 90 days. it was hell for my wife and 4 kids. i look forward to new treatments for stroke spasticity. the md that prescribed the pump for me and medtronics kept telling me it can't be the pump or the baclofen to my family. i was shifted to a chemical dependency psychiatrist who told the doctor that put pump and baclofen in my system to get over to his hospital and shut the dam pump off. soon as that was done i immediately got my sanity back. i should have sued the hell out of the doctor and medtronics for their lack of information on baclofen. hindsight is 20-20. i thought if you sued the medical establishment, that they would blackball you. i look forward to new treatments for treatment of any disease treatments for muscle spasticity. froma doctor who has severe muscle spasticity from a stroke from 16 years ago....

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